CONCLUSIONS: Based on the totality of data on improvements in height-based parameters combined with exposure-response analyses, somapacitan 0.24 mg/kg/week appears most efficacious, providing similar efficacy, safety, and tolerability as daily GH 0.067 mg/kg/day in short children born SGA after 26 weeks of treatment.
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Category: Pediatrics
Long-acting PEGylated growth hormone in children with idiopathic short stature: time to reconsider our diagnostic and treatment policy? by Jan M Wit
Idiopathic short stature (ISS) is a diagnosis of exclusion, and therefore each child with short stature or slow growth referred to a paediatrician deserves a full medical history and physical examination, as well as radiological and laboratory screening tests. In patients with an increased likelihood of a genetic cause, genetic testing is indicated. Idiopathic short…
Microphysiological Drug-testing Platform for Identifying Responses to Prodrug Treatment in Primary Leukemia by Furkan Gökçe
Despite increasing survival rates of pediatric leukemia patients over the past decades, the outcome of some leukemia subtypes has remained dismal. Drug sensitivity and resistance testing on patient-derived leukemia samples provide important information to tailor treatments for high-risk patients. However, currently used well-based drug screening platforms have imitations in predicting the effects of prodrugs, a…
VEGF gene polymorphisms in Iranian patients with intracranial glioblastoma by Sara Hanaei
CONCLUSIONS: Polymorphisms of VEGF could potentially play a role in pathogenesis of GBM, as the allele and genotype distributions of rs3025039 and rs2010963 SNPs were significantly associated with GBM occurrence.
Fiberoptic Endoscopic Evaluation of Swallowing in the Breastfeeding Infant by James W Schroeder
CONCLUSIONS: FEES is the instrumental exam of choice when evaluating a primarily breastfed infant who has suspected dysphagia. The exam is well tolerated and provides accurate, objective information while accounting for this population’s unique swallowing physiology. Primarily breastfed infants presenting with laryngeal congestion are more likely to have clinically worse dysphagia than those presenting with…
Treatment with pollen allergen immunotherapy improves health-related quality of life in children and adolescents: a three-year follow-up-study by Helena Agenäs
CONCLUSION: After 1 year of pollen SCIT, HRQoL improved, and symptoms decreased; these changes were maintained during the study period. The proportion of severe and very severe symptoms significantly decreased.
Ultrasound-guided Versus Computed Tomography Fluoroscopy-assisted Cervical Transforaminal Steroid Injection for the Treatment of Radicular Pain in the Lower Cervical Spine: A Randomized Single-blind Controlled Noninferiority Study by Li Yue
CONCLUSIONS: This study demonstrated that US provided a noninferior injectate spread pattern and similar improvement of radicular pain and functional status when compared with CT-guided TFSI. US may be advantageous during this procedure because it allows visualization of critical vessels and avoids radiation exposure.
KIN: a method to infer relatedness from low-coverage ancient DNA by Divyaratan Popli
Genetic kinship of ancient individuals can provide insights into their culture and social hierarchy, and is relevant for downstream genetic analyses. However, estimating relatedness from ancient DNA is difficult due to low-coverage, ascertainment bias, or contamination from various sources. Here, we present KIN, a method to estimate the relatedness of a pair of individuals from…
The Implications of Airway Anomalies on Children with Congenital Heart Disease and Bronchiolitis by Po-Yuan Wang
BACKGROUND: Bronchiolitis is a common airway infection in young children. Hemodynamically significant congenital heart disease (CHD) predicts a more complicated course. However, the role of airway anomalies remains unknown.
Significance of a multidisciplinary approach to congenital extrahepatic portosystemic shunt; A changing paradigm for the treatment by Hajime Uchida
CONCLUSION: Earlier therapeutic interventions should be strongly considered for patients with POPH related to CEPS. However, in view of the invasiveness and treatment complications, special attention should be paid to the management of patdients with short shunt types. This article is protected by copyright. All rights reserved.
T-replete cord transplants give superior outcomes in high risk and relapsed/refractory paediatric myeloid malignancy by Claire Horgan
Stem cell transplant (SCT) outcomes in high-risk (HR) and relapsed/refractory (R/R) paediatric acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) have been poor historically. Cord blood allows T-cell replete transplant (TRCB), enabling enhanced graft-versus-leukaemia. We collected data from 367 consecutive patients undergoing TRCB (112 patients) or other cell source (255 patients) SCT for paediatric AML/MDS…
Targeting Microglial Metabolic Rewiring Synergizes with Immune Checkpoint Blockade Therapy for Glioblastoma by Zengpanpan Ye
Glioblastoma (GBM) constitutes the most lethal primary brain tumor for which immunotherapy has provided limited benefit. The unique brain immune landscape is reflected in a complex tumor immune microenvironment (TIME) in GBM. Here, single cell sequencing of the GBM TIME revealed that microglia were under severe oxidative stress, which induced nuclear receptor subfamily 4 group…
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